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Scripps Gets NIH Funding for Method for Targeting RNA in the Genome

NEW YORK (GenomeWeb News) – A Scripps Research Institute scientist has received a $1.5 million grant from the National Institutes of Health to develop a computer-based design method for targeting RNA in the human genome in order to discover new therapeutics, Scripps said today.

Scripps Research Assistant Professor Matthew Disney, the principal investigator on the grant, has created a computer program that merges information on the interaction between small molecules and RNA folds in the genome that may contribute to human diseases.

"The rational design of small molecules that exploit therapeutic targets from genomic sequence was one of the promises of the human genome project, but people had no idea of how to do it," Disney said in a statement. "As a result, most targets—especially RNA targets—represent untapped potential."

He explained that the Scripps method subverts the conventional top-down method of screening RNA targets against a chemical library. "Our bottom-up approach uses information about the small RNA motifs such as internal loops or hairpins that a small molecule ligand prefers to bind," Disney said.

He said that he has already used this approach to design small molecules that are active against myotonic dystrophy type 1, the most common type of muscular dystrophy.

Scripps said all of the tools developed through this project will be made available on the internet so that they may be shared by the RNA research community.

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